In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy:: current status; engineered T cells; NK-cell therapy; hemoglobinopathies:: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more.
Evolution of Gene Therapy, Historical Perspective A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System Adeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle Chimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic Malignancies Genome-Edited T Cell Therapies Gene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic Malignancies Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease Hemophilia A/B Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity Gene Therapy for Pediatric Neurologic Disease Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
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